BR112022020423A2 - COMPOUNDS AND METHODS FOR SPLICING MODULATION - Google Patents

COMPOUNDS AND METHODS FOR SPLICING MODULATION

Info

Publication number
BR112022020423A2
BR112022020423A2 BR112022020423A BR112022020423A BR112022020423A2 BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2 BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2
Authority
BR
Brazil
Prior art keywords
compounds
methods
splicing
splicing modulation
modulation
Prior art date
Application number
BR112022020423A
Other languages
Portuguese (pt)
Inventor
Reynolds Dominic
Leger Serge
W Seiler Michael
A Agrawal Anant
Vaillancourt Frederic
Smith Peter
T Hopper Allen
Prajapati Sudeep
Soueidan Olivier
Original Assignee
Remix Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Remix Therapeutics Inc filed Critical Remix Therapeutics Inc
Publication of BR112022020423A2 publication Critical patent/BR112022020423A2/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/502—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with carbocyclic ring systems, e.g. cinnoline, phthalazine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings
    • C07D401/12—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

Abstract

COMPOSTOS E MÉTODOS PARA MODULAÇÃO DE SPLICING. A presente invenção refere-se a compostos e composições relacionadas que, entre outros, modulam o splicing de ácido nucleico, por exemplo, splicing de um pré-mRNA, assim como a métodos de uso dos mesmos.COMPOUNDS AND METHODS FOR SPLICING MODULATION. The present invention relates to compounds and related compositions which, inter alia, modulate nucleic acid splicing, for example, splicing of a pre-mRNA, as well as methods of using the same.

BR112022020423A 2020-04-08 2021-04-08 COMPOUNDS AND METHODS FOR SPLICING MODULATION BR112022020423A2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063007331P 2020-04-08 2020-04-08
US202063044318P 2020-06-25 2020-06-25
US202063072922P 2020-08-31 2020-08-31
US202063126494P 2020-12-16 2020-12-16
PCT/US2021/026481 WO2021207554A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (1)

Publication Number Publication Date
BR112022020423A2 true BR112022020423A2 (en) 2022-11-29

Family

ID=75690721

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022020423A BR112022020423A2 (en) 2020-04-08 2021-04-08 COMPOUNDS AND METHODS FOR SPLICING MODULATION

Country Status (14)

Country Link
US (2) US20230167093A1 (en)
EP (1) EP4132646A1 (en)
JP (1) JP2023520916A (en)
KR (1) KR20220167298A (en)
CN (1) CN115551593A (en)
AU (1) AU2021253587A1 (en)
BR (1) BR112022020423A2 (en)
CA (1) CA3174353A1 (en)
CL (1) CL2022002780A1 (en)
CR (1) CR20220566A (en)
IL (1) IL297137A (en)
MX (1) MX2022012678A (en)
TW (1) TW202208344A (en)
WO (2) WO2021207532A1 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR122020005073A2 (en) 2017-08-04 2020-10-13 Skyhawk Therapeutics, Inc. COMPOUND, PHARMACEUTICAL COMPOSITION AND USE OF THE COMPOUND
JP2023532623A (en) 2020-05-13 2023-07-31 シーエイチディーアイ ファウンデーション，インコーポレーテッド HTT modulators for treating Huntington's disease
CA3233973A1 (en) * 2021-10-13 2023-04-20 Dominic Reynolds Compounds and methods for modulating nucleic acid splicing
WO2023097007A1 (en) * 2021-11-24 2023-06-01 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023133225A1 (en) * 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2024054591A1 (en) 2022-09-07 2024-03-14 Arvinas Operations, Inc. Rapidly accelerated fibrosarcoma (raf) degrading compounds and associated methods of use

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1163237B1 (en) * 1999-03-17 2004-05-06 AstraZeneca AB Amide derivatives
GB0324790D0 (en) * 2003-10-24 2003-11-26 Astrazeneca Ab Amide derivatives
TWI320783B (en) * 2005-04-14 2010-02-21 Otsuka Pharma Co Ltd Heterocyclic compound
BRPI0817061A2 (en) * 2007-09-12 2015-03-24 Wyeth Llc Pyrrolidinylalkylisoquinolinone and -isoindolinoline derivatives as histamine-3 antagonists
TR201813877T4 (en) 2012-02-10 2018-11-21 Hoffmann La Roche COMPOUNDS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY.
MY174339A (en) 2012-08-13 2020-04-09 Novartis Ag 1,4-disubstituted pyridazine analogs and methods for treating smn-deficiency-related conditions
CA2906542A1 (en) * 2013-03-15 2014-09-25 Intellikine, Llc Combination of kinase inhibitors and uses thereof
EP3256126B1 (en) 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
MA41898A (en) * 2015-04-10 2018-02-13 Hoffmann La Roche BICYCLIC QUINAZOLINONE DERIVATIVES
WO2016196386A1 (en) 2015-05-30 2016-12-08 Ptc Therapeutics, Inc. Methods for modulating rna splicing
EP3353164B1 (en) * 2015-09-23 2021-11-03 Janssen Pharmaceutica, N.V. Bi-heteroaryl substituted 1,4-benzodiazepines and uses thereof for the treatment of cancer
LT3386511T (en) 2015-12-10 2021-08-25 Ptc Therapeutics, Inc. Methods for treating huntington`s disease
EP3523292B1 (en) * 2016-10-10 2021-12-15 Dong-A Socio Holdings Co., Ltd. Heteroaryl compounds and their use as mer inhibitors
EA201991309A1 (en) 2016-11-28 2019-11-29 RNA SPLICING MODULATION METHODS
CN109071448B (en) * 2017-01-22 2020-03-17 福建广生堂药业股份有限公司 ASK1 inhibitor and preparation method and application thereof
KR20200017476A (en) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. How to Change RNA Splicing
BR122020005073A2 (en) 2017-08-04 2020-10-13 Skyhawk Therapeutics, Inc. COMPOUND, PHARMACEUTICAL COMPOSITION AND USE OF THE COMPOUND
EP3688187A4 (en) 2017-09-25 2021-09-29 Skyhawk Therapeutics, Inc. Methods and compositions for screening and identification of splicing modulators
CN109721597B (en) * 2017-10-30 2021-05-14 上海迪诺医药科技有限公司 Pyridoazepine derivative, and pharmaceutical composition and application thereof
WO2019094312A1 (en) * 2017-11-08 2019-05-16 Merck Sharp & Dohme Corp. Prmt5 inhibitors
EP3717471B1 (en) * 2017-12-02 2022-01-05 Galapagos NV Novel compounds and pharmaceutical compositions thereof for the treatment of diseases
US11530207B2 (en) 2018-04-10 2022-12-20 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
CR20210050A (en) 2018-06-27 2021-06-10 Reborna Biosciences Inc Prophylactic or therapeutic agent for spinal muscular atrophy

Also Published As

Publication number Publication date
EP4132646A1 (en) 2023-02-15
US20230167093A1 (en) 2023-06-01
CL2022002780A1 (en) 2023-04-28
CR20220566A (en) 2023-01-23
CA3174353A1 (en) 2021-10-14
KR20220167298A (en) 2022-12-20
JP2023520916A (en) 2023-05-22
AU2021253587A1 (en) 2022-12-01
CN115551593A (en) 2022-12-30
IL297137A (en) 2022-12-01
US20230159496A1 (en) 2023-05-25
TW202208344A (en) 2022-03-01
MX2022012678A (en) 2023-01-11
WO2021207554A1 (en) 2021-10-14
WO2021207532A1 (en) 2021-10-14

Similar Documents

Publication Publication Date Title
BR112022017107A2 (en) PYRIDAZINE DERIVATIVES FOR NUCLEIC ACID SPLICING MODULATION
BR112022020423A2 (en) COMPOUNDS AND METHODS FOR SPLICING MODULATION
BR112022020418A2 (en) COMPOUNDS AND METHODS FOR SPLICING MODULATION
BR112022017210A2 (en) COMPOUNDS AND METHODS FOR SPLICING MODULATION
BR112023022592A2 (en) STAPLE CARTRIDGE COMPRISING STAPLE DRIVERS AND STABILITY BRACKETS
BR112021024108A2 (en) Tead inhibitors and uses thereof
CL2020000937A1 (en) Multispecific antibody.
BR112016014020A2 (en) GPR6 TETRAHYDROPYRIDOPYRAZINE MODULATING COMPOUNDS, THEIR SALTS AND THEIR USES
BRPI0811926A8 (en) HERBICIDIAL COMPOSITIONS, PROCESS FOR PREPARING A HERBICIDIAL COMPOSITION, PROCESS FOR CONTROLING UNDESIRABLE VEGETATION AND USE OF A FATTY ACID TO STABILIZE A HERBICIDIAL COMPOSITION
MX2022010681A (en) Compounds and methods for modulating splicing.
BR112023021068A2 (en) COMPOUNDS, COMPOSITIONS AND METHODS FOR TREATING CANCER
BR112018002904A2 (en) high temperature amine stabilized alkylthiophosphoric triamide and / or dcd solvent systems and use in agricultural applications
CR20190150A (en) Highly concentrated low viscosity masp-2 inhibitory antibody formulations, kits, and methods
BR112018075201A2 (en) beta-3 adrenergic receptor modulators useful for treating or preventing related disorders
BR112018070664A2 (en) new gpr119 agonist compounds
BR112022009723A2 (en) SPECIFIC ANTIBODIES TO CD47, PD-L1, AND USES THEREOF
CL2021001461A1 (en) trex1 modulators
BR112019000356A2 (en) compounds and methods for smn2 modulation
CO2023006684A2 (en) Antibodies against sars-cov-2 and their uses
BR112023023223A2 (en) CDK2 DEGRADERS AND USES THEREOF
BR112023018619A2 (en) PLASMA KALYKREIN HETEROARYL INHIBITORS
CR20220483A (en) Compounds and methods for modulating splicing
DOP2020000226A (en) METHOD FOR MARKING A PETROLEUM HYDROCARBON
AR120850A1 (en) MODULATORS OF NECROPTOSIS, SCREENING PROCEDURES AND PHARMACEUTICAL COMPOSITIONS
BR112022000188A2 (en) Method for the preparation of 2-(phenylimino)-1,3-thiazolidin-4-ones